ALS & Neuromuscular Disorders

Select a condition to begin searching our available clinical trials and research studies.

Amyotrophic lateral sclerosis (ALS)

ALS
The purpose of this research is to determine if Nuedexta, an FDA approved and routinely prescribed medication, improves speech and swallowing functions in people with ALS.
ALS
This study will evaluate whether prolonged oral levosimendan can preserve respiratory function more effectively than placebo, resulting in better patient functionality.
ALS
This is a multicenter, multiple dose study to examine the effect of Acthar on functional decline in adult subjects with amyotrophic lateral sclerosis (ALS).
ALS
This study will comprise an 18-week open-label safety and tolerability trial.
ALS
The investigators are interested in the therapeutic potential of acute intermittent hypoxia (AIH) for individuals with neuromuscular diseases, such as ALS.
ALS
The purpose of this study is to investigate the effects of two treatment techniques called Expiratory Muscle Strength Training (EMST) and Lung Volume Recruitment (LVR) on breathing, swallowing, speech, and cough function in persons with mild to moderate ALS.
ALS
The purpose of this study is to evaluate the discriminant ability of several non-invasive screening tools at detecting swallowing impairment in individuals with ALS.
ALS
This registry will ask participants diagnosed with ALS for clinical and demographic data to help researchers discover future treatments.
ALS
The purpose of the Pennant clinical research study is to evaluate the safety, tolerability, and effectiveness of the study drug in slowing down the loss of motor skills or brain function in patients with ALS and to assess whether the study drug can increase an ALS patient’s life expectancy.

Charcot-Marie Tooth

Charcot-Marie Tooth
This is a multicenter, phase 2 study to evaluate the safety, tolerability, pharmacodynamics (PD), efficacy, and pharmacokinetics (PK) of ACE-083 in patients with CMT1 and CMTX, to be conducted in two parts. Part 1 is non-randomized, open-label, dose-escalation and Part 2 is randomized, double-blind, and placebo-controlled.
Charcot-Marie Tooth
This is a multicenter, randomised, placebo-controlled phase III study (CLN-PXT3003-02) to assess the efficacy and safety of PXT3003. The intermediate and high dose of PXT3003 demonstrated an improvement of disability in this patient population in the phase II study.

Muscular dystrophy

Muscular dystrophy
The purpose of this research study is to determine how accurate different tests are at identifying swallowing difficulties in persons with OPMD.
Muscular dystrophy
This study aims to help drug developers successfully design clinical trials and understand how to assess the efficacy of potential therapies. Researchers are trying to learn more about what causes the muscle weakness and stiffness (myotonia) in myotonic dystrophy.
Muscular dystrophy
This study is designed to obtain data regarding 2 aspects of the phenotype in myotonic dystrophy (dystrophia myotonica or DM). These are multi-system diseases leading to symptoms in many regions of the body including skeletal muscles, central nervous system and the GI tract.
Muscular dystrophy
This study is designed to assess social cognition deficits in adult onset patients with myotonic dystrophy type 1 and understand possible neuroimaging correlates of such deficits.